JP2024529108A - スタスミン2(stmn2)を標的とするrnaガイドを含む遺伝子編集システム及びその使用 - Google Patents
スタスミン2(stmn2)を標的とするrnaガイドを含む遺伝子編集システム及びその使用 Download PDFInfo
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- JP2024529108A JP2024529108A JP2024508332A JP2024508332A JP2024529108A JP 2024529108 A JP2024529108 A JP 2024529108A JP 2024508332 A JP2024508332 A JP 2024508332A JP 2024508332 A JP2024508332 A JP 2024508332A JP 2024529108 A JP2024529108 A JP 2024529108A
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C12N15/09—Recombinant DNA-technology
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0684—Cells of the urinary tract or kidneys
- C12N5/0686—Kidney cells
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2800/00—Nucleic acids vectors
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Health & Medical Sciences (AREA)
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- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
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- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Urology & Nephrology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Cell Biology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hospice & Palliative Care (AREA)
- Virology (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Mycology (AREA)
- Marine Sciences & Fisheries (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Crystallography & Structural Chemistry (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163231784P | 2021-08-11 | 2021-08-11 | |
| US63/231,784 | 2021-08-11 | ||
| US202263322002P | 2022-03-21 | 2022-03-21 | |
| US63/322,002 | 2022-03-21 | ||
| PCT/US2022/040042 WO2023018858A1 (en) | 2021-08-11 | 2022-08-11 | Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024529108A true JP2024529108A (ja) | 2024-08-01 |
| JP2024529108A5 JP2024529108A5 (https=) | 2025-08-19 |
Family
ID=83193363
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024508332A Pending JP2024529108A (ja) | 2021-08-11 | 2022-08-11 | スタスミン2(stmn2)を標的とするrnaガイドを含む遺伝子編集システム及びその使用 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20230203539A1 (https=) |
| EP (1) | EP4384618A1 (https=) |
| JP (1) | JP2024529108A (https=) |
| KR (1) | KR20240052763A (https=) |
| AU (1) | AU2022328401A1 (https=) |
| CA (1) | CA3228487A1 (https=) |
| CO (1) | CO2024002315A2 (https=) |
| IL (1) | IL310616A (https=) |
| MX (1) | MX2024001878A (https=) |
| WO (1) | WO2023018858A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023205844A1 (en) * | 2022-04-26 | 2023-11-02 | Peter Maccallum Cancer Institute | Nucleic acids and uses thereof |
| KR20250151430A (ko) * | 2023-02-15 | 2025-10-21 | 아버 바이오테크놀로지스, 인크. | 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019178427A1 (en) | 2018-03-14 | 2019-09-19 | Arbor Biotechnologies, Inc. | Novel crispr dna targeting enzymes and systems |
| CA3126918A1 (en) * | 2019-01-14 | 2020-07-23 | President And Fellows Of Harvard College | Methods and compositions for restoring stmn2 levels |
| EP3976010A4 (en) * | 2019-06-03 | 2023-08-02 | Quralis Corporation | Oligonucleotides and methods of use for treating neurological diseases |
| CN121380024A (zh) | 2020-03-31 | 2026-01-23 | 阿伯生物技术公司 | 包含Cas12i2变体多肽的组合物及其用途 |
| CN115851665B (zh) * | 2021-05-27 | 2025-01-24 | 中国科学院动物研究所 | 工程化的Cas12i核酸酶及其效应蛋白以及用途 |
-
2022
- 2022-08-11 US US17/885,876 patent/US20230203539A1/en not_active Abandoned
- 2022-08-11 CA CA3228487A patent/CA3228487A1/en active Pending
- 2022-08-11 AU AU2022328401A patent/AU2022328401A1/en active Pending
- 2022-08-11 KR KR1020247007547A patent/KR20240052763A/ko active Pending
- 2022-08-11 EP EP22765309.4A patent/EP4384618A1/en active Pending
- 2022-08-11 WO PCT/US2022/040042 patent/WO2023018858A1/en not_active Ceased
- 2022-08-11 MX MX2024001878A patent/MX2024001878A/es unknown
- 2022-08-11 US US18/682,643 patent/US20250127924A1/en active Pending
- 2022-08-11 IL IL310616A patent/IL310616A/en unknown
- 2022-08-11 JP JP2024508332A patent/JP2024529108A/ja active Pending
-
2024
- 2024-02-27 CO CONC2024/0002315A patent/CO2024002315A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA3228487A1 (en) | 2023-02-16 |
| CO2024002315A2 (es) | 2024-03-07 |
| WO2023018858A1 (en) | 2023-02-16 |
| US20250127924A1 (en) | 2025-04-24 |
| MX2024001878A (es) | 2024-02-29 |
| IL310616A (en) | 2024-04-01 |
| US20230203539A1 (en) | 2023-06-29 |
| KR20240052763A (ko) | 2024-04-23 |
| AU2022328401A1 (en) | 2024-02-22 |
| EP4384618A1 (en) | 2024-06-19 |
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